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Establishment of the Effectiveness of Early Versus Late Stem Cell Gene Therapy in Mucopolysaccharidosis II for Treating Central Versus Peripheral Disease.
Mandolfo, Oriana; Liao, Aiyin; Singh, Esha; O'leary, Claire; Holley, Rebecca J; Bigger, Brian W.
Affiliation
  • Mandolfo O; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom.
  • Liao A; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom.
  • Singh E; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom.
  • O'leary C; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom.
  • Holley RJ; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom.
  • Bigger BW; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom.
Hum Gene Ther ; 35(7-8): 243-255, 2024 Apr.
Article in En | MEDLINE | ID: mdl-37427450
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Mucopolysaccharidosis II / Medically Unexplained Symptoms / Iduronate Sulfatase / Nervous System Diseases Type of study: Prognostic_studies Limits: Animals / Child / Humans / Infant Language: En Journal: Hum Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2024 Document type: Article Affiliation country: Reino Unido Country of publication: Estados Unidos
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Mucopolysaccharidosis II / Medically Unexplained Symptoms / Iduronate Sulfatase / Nervous System Diseases Type of study: Prognostic_studies Limits: Animals / Child / Humans / Infant Language: En Journal: Hum Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2024 Document type: Article Affiliation country: Reino Unido Country of publication: Estados Unidos